Introduction: Hepatocellular carcinoma (HCC) is an aggressive primary tumor of the liver. It mostly occurs in patients with chronic liver disease. No effective systemic treatment is available for patients who progress on or are intolerant to sorafenib. The complexity of HCC, driven by multiple pathways and growth factor systems, is increased by the underlying cirrhosis, and by the lack of validated prognostic factors. Successful HCC therapy requires inhibition of the right target and an optimal drug safety profile. Areas covered: Tivantinib, an oral inhibitor of the hepatocyte growth factor receptor (MET), demonstrated promising antitumor activity in patients with HCC. We reviewed published clinical data on tivantinib for the second-line treatment of HCC. A randomized Phase II trial showed improved overall survival in patients with MET-high tumors. An ongoing Phase III study is investigating tivantinib in patients with advanced, pretreated, MET-high HCC. Expert opinion: Tivantinib significantly increased survival in patients with MET-high HCC. Many clinical trials with systemic therapy did not show a benefit in HCC, and second-line treatment remains a high unmet medical need. If the Phase III trial confirms the results obtained in Phase II, tivantinib will be the standard second-line treatment in patients with MET-high HCC.
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